Lecouturier Jan, Sheerin Neil
Population and Health Sciences Institute, Newcastle University, Newcastle upon Tyne, UK.
National Renal Complement Therapeutic Centre and Newcastle University Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, UK.
Patient. 2025 Sep 12. doi: 10.1007/s40271-025-00771-5.
Atypical haemolytic uremic syndrome (aHUS) is a rare life-threatening disease. Lifelong treatment with intravenous eculizumab every 2/3 weeks was recommended, but evidence is emerging that many patients can stop complement inhibition and restart should they relapse. However, little is known about the opinions and needs of patients with aHUS on withdrawal.
We aimed to understand the factors that affect decisions to withdraw from treatment.
This was a qualitative study embedded in an eculizumab withdrawal trial. Data were collected through in-depth telephone interviews from January 2019 to June 2022. We conducted a thematic analysis using a constant comparative method. Interviewees included eight adults and the parents of 12 children with aHUS approached to participate in the trial. Follow-up interviews were conducted between January 2021 and June 2024 with those who withdrew from eculizumab.
The onset of aHUS had been traumatic for most. Regarding eculizumab, withdrawal group participants talked of the disruptive treatment regimen and side effects, the time off work/school, and impacts on taking holidays. Decisions to withdraw from eculizumab were driven by the wish to lead more normal lives and concerns about long-term treatment. Drivers for declining withdrawal focused on relapse and its perceived impact. After 2 years, the withdrawal group had regained a semblance of normality, though fears about relapse remained, and they were aware of the need for long-term follow-up. Participants had a greater sense of control over the necessary steps should they/their child relapse.
An understanding of patient/parent experiences should guide discussions about eculizumab withdrawal. Although the availability of an alternative complement inhibitor reduces the frequency of infusions, most of the factors influencing withdrawal decisions remain unchanged. Support to alleviate fears in the early stages of withdrawal would be beneficial. Evidence from the main trial on successful withdrawal, and recovery time where eculizumab was reinstated, may provide reassurance to those who are uncertain about withdrawal.
非典型溶血性尿毒症综合征（aHUS）是一种罕见的危及生命的疾病。此前建议每2/3周静脉注射依库珠单抗进行终身治疗，但越来越多的证据表明，许多患者在复发时可以停止补体抑制治疗并重新开始用药。然而，对于aHUS患者在停药方面的意见和需求却知之甚少。
我们旨在了解影响停药决策的因素。
这是一项纳入依库珠单抗撤药试验的定性研究。2019年1月至2022年6月通过深度电话访谈收集数据。我们采用持续比较法进行主题分析。受访者包括8名成年患者以及12名参与试验的aHUS患儿的家长。2021年1月至2024年6月，对那些停用依库珠单抗的患者进行了随访访谈。
对大多数人来说，aHUS的发病是一场创伤。对于依库珠单抗，撤药组参与者谈到了治疗方案的干扰性和副作用、误工/休学情况以及对度假的影响。停药的决定是出于想要过上更正常生活的愿望以及对长期治疗的担忧。拒绝停药的原因主要集中在复发及其可能产生的影响上。两年后，撤药组虽然仍对复发心存恐惧，且意识到需要长期随访，但已恢复了一定程度的正常生活。参与者对自己或孩子复发时应采取的必要措施有了更强的掌控感。
了解患者/家长的经历应有助于指导关于依库珠单抗撤药的讨论。尽管有替代补体抑制剂可减少输液频率，但影响停药决策的大多数因素仍未改变。在撤药早期提供支持以减轻恐惧会很有帮助。主要试验中关于成功撤药以及重新使用依库珠单抗后的恢复时间的证据，可能会让那些对撤药不确定的人安心。
